WORLDSymposium 2025 New Treatment Award

WORLDSymposium strives to recognize important achievements in therapy for lysosomal diseases for treatments attaining regulatory approval. In recognition of achieving major milestones, WORLDSymposium reviews potential recipients for the New Treatment Award in 2025. This award honors “new treatments” that are viewed as providing value to patients with lysosomal diseases, and general acceptance as evidenced by approval by the U.S. Food and Drug Administration, European Medicines Agency, and other regulatory authorities.

Nominations for this award will be accepted from July 15 to October 31, 2024, and may be submitted for any therapy achieving regulatory approval since the last New Treatment Award was bestowed in 2024.

The WORLDSymposium 2025 New Treatment Awards will be presented on Friday, February 7, 2025, at 08:00 PST, followed by a full day of Late-breaking Science and Contemporary Forum abstract presentations.

Past Award Recipients:

2024 cipaglucosidase alfa (Pombiliti®, Amicus Therapeutics) and and miglustat (Opfolda®, Amicus Therapeutics), pegunigalsidase alfa-iwxj (Elfabrio®, Chiesi Global Rare Diseases), velmanase alfa-tycv (Lamzede®, Chiesi Global Rare Diseases) 
2023 olipudase alfa-rpcp (Xenpozyme™, Sanofi) 
2022 avalglucosidase alfa-ngpt (Nexviazyme®, Sanofi Genzyme)
2022 pabinafusp alfa (IZCARGO®, JCR Pharmaceuticals)
2018 cerliponase alfa (Brineura™, BioMarin)
2018 vestronidase alfa (MEPSEVII™, Ultragenyx)
2017 migalastat (Galafold™, Amicus)
2016 sebelipase alfa (Kanuma™, Alexion)
2015 eliglustat (Cerdelga™, Sanofi Genzyme)
2015 idursulfase beta (Hunterase®, GC Pharma)
2015 cysteamine bitartrate (PROCYSBI™, Horizon Therapeutics)
2015 elosulfase alfa (Vimizim®,  BioMarin)